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• Response to eletter
  Ravi Banthia
  Published on: 15 April 2021
• Selumetinib should be preferred to a surgical approach in case of extensive plexiform neurofibromas
  Francesco Baldo, Laura Morra, Andrea Magnolato and Egidio Barbi
  Published on: 22 November 2020

• Published on: 15 April 2021

  Response to eletter

  • Ravi Banthia, Doctor Urology and Renal Transplantation, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, Uttar Pradesh, India

  Thanks for the comments on our manuscript entitled "Plexiform neurofibromatosis of penis: a rare presentation of type 1 neurofibromatosis."

   

  We think that this is a very good suggestion for treating such cases. Selumetinib has been found to be effective to treat neurofibromatosis type 1 in children 2 years of age and older. It is an inhibitor of mitogen-activated protein kinase and has been recommended as a first-line therapy approved for paediatric neurofibromatosis patients who have inoperable and bulky lesions. 

  Selumetinib therapy was a good option for this particular child but there were several reasons to choose surgery for this patient. Firstly the deformity was unsightly and grotesque considering the almost double length of the penis was leading to social discrimination, peer pressure and solitary life for this child. The patient has been rehabilitated with just one surgical operation in which after debulking the penile size is within socially acceptable limits. S...

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  Thanks for the comments on our manuscript entitled "Plexiform neurofibromatosis of penis: a rare presentation of type 1 neurofibromatosis."

   

  We think that this is a very good suggestion for treating such cases. Selumetinib has been found to be effective to treat neurofibromatosis type 1 in children 2 years of age and older. It is an inhibitor of mitogen-activated protein kinase and has been recommended as a first-line therapy approved for paediatric neurofibromatosis patients who have inoperable and bulky lesions. 

  Selumetinib therapy was a good option for this particular child but there were several reasons to choose surgery for this patient. Firstly the deformity was unsightly and grotesque considering the almost double length of the penis was leading to social discrimination, peer pressure and solitary life for this child. The patient has been rehabilitated with just one surgical operation in which after debulking the penile size is within socially acceptable limits. Secondly, there were financial issues in procuring the drug as the treatment is expensive considering the high monthly costs that are involved in therapy with this particular drug. Thirdly medical treatment with this drug if available would have taken some time for the lesion to regress and the patient's parents were looking for a treatment that could immediately restore normalcy to the deformity. 

   

  We will definitely consider drug therapy with Selumetinib for this patient in the follow-up period as it has been shown to be quite effective in treating extensive neurofibromatosis as was seen in this paediatric patient.

   

  Best Regards,

  Ravi Banthia

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  Conflict of Interest:
  None declared.

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• Published on: 22 November 2020

  Selumetinib should be preferred to a surgical approach in case of extensive plexiform neurofibromas

  • Francesco Baldo, Pediatric Resident University of Trieste
  • Other Contributors:
    • Laura Morra, Pediatric Resident
    • Andrea Magnolato, Pediatrician
    • Egidio Barbi, Pediatrician

  Dear Editor,
  we found the case report of Banthia et al. extremely interesting. First of all, penile plexiform neurofibromas are quite infrequent. Secondly, the subject described did not have a former clinical diagnosis of neurofibromatosis type I prior to the identification of this tumor, which is even rarer. However, we take exception to the therapeutic approach chosen by the authors. In fact, the surgical treatment of deep and extended plexiform neurofibromas is generally unsatisfactory, since their complete resection is frequently unattainable, allowing the masses to grow back (1-2). A partial tumor debulking should be considered only in case of high-risk conditions, such as a urethral or ureteral compression or a bowel obstruction. Apart from these scenarios, a medical approach should always be preferred, at least as a first attempt.
  Selumetinib, an inhibitor of MEK 1 and 2 kinase, has demonstrated to be effective in reducing the size of plexiform neurofibromas in pediatric patients (3-4). The drug is generally well tolerated and safe in the pediatric population. Only few patients failed to show a clinical response to the treatment and even fewer had to stop it due to the appearance of severe adverse events.
  In a recent study, we reported a cohort of nine patients with inoperable plexiform neurofibromas treated with selumetinib (5). Eight of them showed a partial response to the drug, i.e. a reduction of the tumor size by more than 20%. Remarkably, on...

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  Dear Editor,
  we found the case report of Banthia et al. extremely interesting. First of all, penile plexiform neurofibromas are quite infrequent. Secondly, the subject described did not have a former clinical diagnosis of neurofibromatosis type I prior to the identification of this tumor, which is even rarer. However, we take exception to the therapeutic approach chosen by the authors. In fact, the surgical treatment of deep and extended plexiform neurofibromas is generally unsatisfactory, since their complete resection is frequently unattainable, allowing the masses to grow back (1-2). A partial tumor debulking should be considered only in case of high-risk conditions, such as a urethral or ureteral compression or a bowel obstruction. Apart from these scenarios, a medical approach should always be preferred, at least as a first attempt.
  Selumetinib, an inhibitor of MEK 1 and 2 kinase, has demonstrated to be effective in reducing the size of plexiform neurofibromas in pediatric patients (3-4). The drug is generally well tolerated and safe in the pediatric population. Only few patients failed to show a clinical response to the treatment and even fewer had to stop it due to the appearance of severe adverse events.
  In a recent study, we reported a cohort of nine patients with inoperable plexiform neurofibromas treated with selumetinib (5). Eight of them showed a partial response to the drug, i.e. a reduction of the tumor size by more than 20%. Remarkably, one of them, a five-year-old boy, had a giant plexiform neurofibroma similar to the one described in this clinical case, extended from the abdomen to the pelvis and scrotum. The mass was causing abdominal and scrotal pain, so that the boy couldn’t even sit on a chair. The patient had originally been treated in another institution with a partial debulking procedure, but after twelve months the plexiform neurofibroma grew back to its original size. Furthermore, the surgical excision resulted in the necessity of placing a colostomy, which the boy still has and that will probably be impossible to remove in future, since the plexiform neurofibroma further grew around the distal colic stump. After two years of treatment with selumetinib, a radiological decrease of the mass by more than 20% was noticed, and the boy stopped complaining of abdominal and scrotal pain. Nowadays he’s 10 years old and in good clinical conditions.
  In conclusion, we suggest that selumetinib should be preferred to a surgical approach in presence of an extended plexiform neurofibroma, due to the well-known risk or the tumor regrowth after surgery.

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  Conflict of Interest:
  None declared.

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